Davidson, A. I., Halstead, S. K., Goodfellow, J. A., Chavada, G., Mallik, A., Overell, J., Lunn, M. P., McConnachie, A. , van Doorn, P. and Willison, H. J. (2017) Inhibition of complement in Guillain-Barré Syndrome: the ICA-GBS study. Journal of the Peripheral Nervous System, 6, pp. 4-12. (doi: 10.1111/jns.12194) (PMID:27801990)
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Abstract
The outcome of Guillain-Barré syndrome remains unchanged since plasma exchange and intravenous immunoglobulin were introduced over 20 years ago. Pathogenesis studies on GBS have identified the terminal component of complement cascade as a key disease mediator and therapeutic target. We report the first use of terminal complement pathway inhibition with eculizumab in humans with GBS. In a randomised, double-blind, placebo-controlled trial, 28 subjects eligible on the basis of GBS disability grade of at least 3 were screened, of whom 8 (29%) were randomised. Five received eculizumab for four weeks, alongside standard intravenous immunoglobulin treatment. The safety outcomes, monitored via adverse events capture, showed eculizumab to be well tolerated and safe when administered in conjunction with IVIg. Primary and secondary efficacy outcomes in the form of GBS disability scores, MRC sum scores, Rasch Overall Disability Scores and Overall Neuropathy Limitation Scores are reported descriptively. For the primary efficacy outcome at 4 weeks after recruitment, 2 of 2 placebo and 2 of 5 eculizumab-treated subjects had improved by 1 or more grades on the GBS disability score. Although the small sample size precludes a statistically meaningful analysis, these pilot data indicate further studies on complement inhibition in GBS are warranted.
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